In a land­mark first, Chi­nese sci­en­tist claims birth of ge­net­i­cal­ly mod­i­fied ba­bies — and all hell breaks loose

A Chi­nese re­searcher is claim­ing that he cre­at­ed the first-ever ge­net­i­cal­ly edit­ed ba­bies us­ing CRISPR/Cas9 tools that have now be­come a com­mon fea­ture in labs around the world. And the news trig­gered a tem­pest in sci­en­tif­ic cir­cles the world over as re­searchers who have been us­ing gene-edit­ing tech to re­fine plants and forge new ther­a­pies try to puz­zle out the stun­ning —if true — de­vel­op­ment fea­tured on YouTube.

Sev­er­al ex­perts — in­clud­ing co-in­ven­tor Feng Zhang — crit­i­cized the ex­per­i­ment, rais­ing po­ten­tial safe­ty is­sues that could arise as a re­sult of the ge­net­ic tin­ker­ing. And Rice Uni­ver­si­ty, where a pro­fes­sor was re­port­ed­ly in­volved, is in­ves­ti­gat­ing.

The sci­en­tist who claimed cred­it for the work, though, of­fered a sun­ny per­spec­tive on YouTube.

“Two beau­ti­ful Chi­nese girls named Lu­lu and Nana came cry­ing in­to the world as healthy as any oth­er ba­bies a few weeks ago,” said Shen­zhen-based re­searcher Jiankui He in the YouTube video and WeChat post. But this was no av­er­age birth. The re­searcher’s un­ver­i­fied claim is that the twins’ em­bryos have been ge­net­i­cal­ly en­gi­neered with CRISPR to de­com­mis­sion CCR5, a gene used by HIV as a back door in­to a cell.

Ahead of an in­ter­na­tion­al con­fer­ence on gene edit­ing ex­pect­ed to com­mence on Tues­day in Hong Kong, He said he had al­tered em­bryos for sev­en cou­ples dur­ing fer­til­i­ty treat­ments, with one preg­nan­cy re­sult­ing thus far. But the le­git­i­ma­cy of the project is be­ing in­ves­ti­gat­ed. The South­ern Uni­ver­si­ty of Sci­ence and Tech­nol­o­gy in Shen­zhen, with which He is af­fil­i­at­ed, is­sued a state­ment say­ing it was “deeply shocked” and un­aware of his re­search project which they con­sid­er a “se­ri­ous vi­o­la­tion of aca­d­e­m­ic ethics and stan­dards.” The uni­ver­si­ty added He has been on leave with­out pay since Feb­ru­ary.

He has claimed his aim was not to cure or pre­vent an in­her­it­ed dis­ease, but to con­fer a trait that is nat­u­ral­ly com­mon in parts of North­ern Eu­rope — an abil­i­ty to re­sist an HIV in­fec­tion from the AIDS virus. But the choice to ed­it this CCR5 gene, the block­ade of which may al­so be ef­fec­tive in thwart­ing cholera and small­pox, im­me­di­ate­ly trig­gered an on­line up­roar over the use of CRISPR to al­ter DNA in a way that could be passed down for gen­er­a­tions to come.

MIT Tech­nol­o­gy Re­view’s An­to­nio Re­gal­a­do point­ed out this could be par­tic­u­lar­ly con­tro­ver­sial be­cause there are eas­i­er and cheap­er ways to pre­vent HIV in­fec­tion, or in­deed sup­press it. Edit­ing em­bryos dur­ing IVF will al­so be ex­pen­sive and in­volve tech­nol­o­gy out of reach for poor­er pock­ets of the world where HIV is ubiq­ui­tous.

Some are al­so sug­gest­ing the sto­ry rais­es oth­er thorny ques­tions. Im­pe­r­i­al Col­lege in­ves­ti­ga­tor Tom El­lis not­ed:

Al­though there is sci­en­tif­ic con­sen­sus that gene edit­ing should not be em­ployed to make “de­sign­er ba­bies” en­dowed with en­hanced phys­i­cal fea­tures or in­tel­lec­tu­al traits, the ju­ry is out on to what de­gree sci­ence should in­ter­fere with na­ture to pre­vent, treat or cure dis­ease.

In ad­di­tion to Chi­na, lab­o­ra­to­ry re­search is un­der­way in Swe­den and the UK, in­ves­ti­gat­ing the po­ten­tial of gene-edit­ing in hu­man em­bryos. But in the Unit­ed States it’s a po­lit­i­cal­ly charged propo­si­tion that has won the nar­row en­dorse­ment of the Na­tion­al Acad­e­my of Sci­ences, which last year rec­om­mend­ed that germ-line mod­i­fi­ca­tion of hu­mans was jus­ti­fied in some cir­cum­stances, such as pre­vent­ing the birth of chil­dren with se­ri­ous dis­eases. This rec­om­men­da­tion will like­ly fall on deaf ears, as sec­tions of the pub­lic ve­he­ment­ly op­pose such in­ter­fer­ence on re­li­gious grounds. In fact such mod­i­fi­ca­tions are prac­ti­cal­ly out of the ques­tion — with laws in place pro­hibit­ing the FDA from even con­sid­er­ing pro­pos­als to cre­ate ge­net­i­cal­ly-edit­ed off­spring.

The promise of CRISPR/Cas9 edit­ing has long been her­ald­ed. How­ev­er, ex­per­i­men­ta­tion with the pro­ce­dure has yield­ed sig­nif­i­cant safe­ty con­cerns. Da­ta pre­sent­ed ear­li­er this year sug­gest that the tool, which is es­sen­tial­ly a pair of mol­e­c­u­lar scis­sors, may in­ad­ver­tent­ly in­crease can­cer risk in some cells, or in­tro­duce ac­ci­den­tal mu­ta­tions — is­sues that could ham­per the de­vel­op­ment of gene-edit­ing ther­a­pies cham­pi­oned by com­pa­nies such as CRISPR Ther­a­peu­tics $CR­SP, Ed­i­tas Med­i­cine $ED­IT and In­tel­lia Ther­a­peu­tics $NT­LA.

“The ge­net­ic edit­ing of a speck-size hu­man em­bryo car­ries sig­nif­i­cant risks, in­clud­ing the risks of in­tro­duc­ing un­want­ed mu­ta­tions or yield­ing a ba­by whose body is com­posed of some edit­ed and some unedit­ed cells. Da­ta on the Chi­nese tri­al site in­di­cate that one of the fe­tus­es is a ‘mo­sa­ic’ of cells that had been edit­ed in dif­fer­ent ways,” Re­gal­a­do un­der­scored in his ar­ti­cle.

He’s project in­volved cou­ples in which the men had HIV but the women did not, and the goal was to pre­vent their chil­dren from suf­fer­ing the same fate.

Ac­cord­ing to the AP re­port, He said that in one twin, both copies of the in­tend­ed gene had been al­tered, while in the oth­er twin, just one copy had been dis­abled — and that there was no ev­i­dence of harm to any oth­er genes. Hu­mans with one copy of the gene can still be in­fect­ed with HIV.

The edit­ing oc­curred dur­ing IVF — first, the sperm was sep­a­rat­ed from the se­men where HIV is known to linger. Then, a sin­gle sperm was placed in­to a soli­tary egg to cre­ate an em­bryo when the gene-edit­ing tool was em­ployed. Cou­ples re­cruit­ed to the study were giv­en free fer­til­i­ty treat­ment in re­turn for their par­tic­i­pa­tion and of­fered the choice to use ei­ther edit­ed or non-edit­ed em­bryos for preg­nan­cy at­tempts. Over­all, 16 of 22 em­bryos were edit­ed, and 11 em­bryos were used in 6 im­plant at­tempts be­fore the twin preg­nan­cy was re­al­ized, He told the AP.

He, who is al­so a founder of a DNA se­quenc­ing com­pa­ny Di­rect Ge­nomics, ob­tained in­formed con­sent from par­tic­i­pants call­ing the study an “AIDS vac­cine de­vel­op­ment project.” How­ev­er, in his ap­pli­ca­tion form seek­ing eth­i­cal ap­proval, he dubbed it a CCR5 gene edit­ing project.

Press re­ports tied his work to Rice Uni­ver­si­ty’s Michael Deem, who now will have to an­swer for what, ex­act­ly, they did. Rice Uni­ver­si­ty was quick to launch its own probe. They not­ed:

Re­cent press re­ports de­scribe a case of ge­nom­ic edit­ing of hu­man em­bryos in Chi­na. These re­ports in­clude a de­scrip­tion of in­volve­ment by Dr. Michael Deem, a pro­fes­sor of bio­engi­neer­ing at Rice Uni­ver­si­ty. This re­search rais­es trou­bling sci­en­tif­ic, le­gal and eth­i­cal ques­tions.  Rice of­fers the fol­low­ing state­ment:

  1. Rice had no knowl­edge of this work.

  2. To Rice’s knowl­edge, none of the clin­i­cal work was per­formed in the Unit­ed States.

  3. Re­gard­less of where it was con­duct­ed, this work as de­scribed in press re­ports, vi­o­lates sci­en­tif­ic con­duct guide­lines and is in­con­sis­tent with eth­i­cal norms of the sci­en­tif­ic com­mu­ni­ty and Rice Uni­ver­si­ty.

  4. We have be­gun a full in­ves­ti­ga­tion of Dr. Deem’s in­volve­ment in this re­search.

And two founders of CRISPR were al­so quick to note their own prob­lems with the Chi­na em­bryo project.

The Broad In­sti­tute’s Feng Zhang, a co-in­ven­tor of the tech­nol­o­gy, had this to say:

Al­though I ap­pre­ci­ate the glob­al threat posed by HIV, at this stage, the risks of edit­ing em­bryos to knock out CCR5 seem to out­weigh the po­ten­tial ben­e­fits, not to men­tion that knock­ing out of CCR5 will like­ly ren­der a per­son much more sus­cep­ti­ble for West Nile Virus. Just as im­por­tant, there are al­ready com­mon and high­ly-ef­fec­tive meth­ods to pre­vent trans­mis­sion of HIV from a par­ent to an un­born child.

Giv­en the cur­rent ear­ly state of genome edit­ing tech­nol­o­gy, I’m in fa­vor of a mora­to­ri­um on im­plan­ta­tion of edit­ed em­bryos, which seems to be the in­ten­tion of the CCR5 tri­al, un­til we have come up with a thought­ful set of safe­ty re­quire­ments first.

Not on­ly do I see this as risky, but I am al­so deeply con­cerned about the lack of trans­paren­cy sur­round­ing this tri­al.


Im­age: Jiankui He. THE HE LAB via YOUTUBE

Nick Leschly via Getty

UP­DAT­ED: Blue­bird shares sink as an­a­lysts puz­zle out $1.8M stick­er shock and an un­ex­pect­ed de­lay

Blue­bird bio $BLUE has un­veiled its price for the new­ly ap­proved gene ther­a­py Zyn­te­glo (Lenti­Glo­bin), which came as a big sur­prise. And it wasn’t the on­ly un­ex­pect­ed twist in to­day’s sto­ry.

With some an­a­lysts bet­ting on a $900,000 price for the β-tha­lassemia treat­ment in Eu­rope, where reg­u­la­tors pro­vid­ed a con­di­tion­al ear­ly OK, blue­bird CEO Nick Leschly said Fri­day morn­ing that the pa­tients who are suc­cess­ful­ly treat­ed with their drug over 5 years will be charged twice that — $1.8 mil­lion — on the con­ti­nent. That makes this drug the sec­ond most ex­pen­sive ther­a­py on the plan­et, just be­hind No­var­tis’ new­ly ap­proved Zol­gens­ma at $2.1 mil­lion, with an­a­lysts still wait­ing to see what kind of pre­mi­um can be had in the US.

Ted Love. HAVERFORD COLLEGE

Glob­al Blood Ther­a­peu­tics poised to sub­mit ap­pli­ca­tion for ac­cel­er­at­ed ap­proval, with new piv­otal da­ta on its sick­le cell dis­ease drug

Global Blood Therapeutics is set to submit an application for accelerated approval in the second-half of this year, after unveiling fresh data from a late-stage trial that showed just over half the patients given the highest dose of its experimental sickle cell disease drug experienced a statistically significant improvement in oxygen-wielding hemoglobin, meeting the study's main goal.

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News­mak­ers at #EHA19: Re­gen­eron, Ar­Qule track progress on re­sponse rates

Re­gen­eron’s close­ly-watched bis­pe­cif­ic con­tin­ues to ring up high re­sponse rates

Re­gen­eron’s high-pro­file bis­pe­cif­ic REGN1979 is back in the spot­light at the Eu­ro­pean Hema­tol­ogy As­so­ci­a­tion sci­en­tif­ic con­fab. And while the stel­lar num­bers we saw at ASH have erod­ed some­what as more blood can­cer pa­tients are eval­u­at­ed, the re­sponse rates for this CD3/CD20 drug re­main high.

A to­tal of 13 out of 14 fol­lic­u­lar lym­phomas re­spond­ed to the drug, a 93% ORR, down from 100% at the last read­out. In 10 out of 14, there was a com­plete re­sponse. In dif­fuse large B-cell lym­phoma the re­sponse rate was 57% among pa­tients treat­ed at the 80 mg to 160 mg dose range. They were all com­plete re­spons­es. And 2 of these Cars were for pa­tients who had failed CAR-T ther­a­py.

Neil Woodford, Woodford Investment Management via YouTube

Un­der siege, in­vest­ment man­ag­er Wood­ford faces an­oth­er in­vest­ment shock

Em­bat­tled UK fund man­ag­er Neil Wood­ford — who has con­tro­ver­sial­ly blocked in­vestors from pulling out from his flag­ship fund to stem the blood­let­ting, af­ter a slew of dis­ap­point­ed in­vestors fled fol­low­ing a se­ries of sour bets — is now pay­ing the price for his ac­tions via an in­vestor ex­o­dus on an­oth­er fund.

Har­g­reaves Lans­down, which has in the past sold and pro­mot­ed the Wood­ford funds via its re­tail in­vest­ment plat­form, has re­port­ed­ly with­drawn £45 mil­lion — its en­tire po­si­tion — from the in­vest­ment man­ag­er’s In­come Fo­cus Fund.

Gene ther­a­pies seize the top of the list of the most ex­pen­sive drugs on the plan­et — and that trend has just be­gun

Anyone looking for a few simple reasons why the gene therapy field has caught fire with the pharma giants need only look at the new list of the 10 most expensive therapies from GoodRx.

Two recently approved gene therapies sit atop this list, with Novartis’ Zolgensma crowned the king of the priciest drugs at $2.1 million. Right below is Luxturna, the $850,000 pioneer from Spark, which Roche is pushing hard to acquire as it adds a gene therapy group to the global mix.

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Fol­low­ing CAR-T pi­o­neer­s' foot­steps, Tes­sa launch­es Chi­na JV in $120M deal

These days just about every biotech se­ri­ous about glob­al de­vel­op­ment — and not just com­mer­cial­iza­tion — has a Chi­na strat­e­gy. Tes­sa Ther­a­peu­tics, a Bay­lor as­so­ci­at­ed out­fit based out of Sin­ga­pore, is no ex­cep­tion.

Tak­ing a page out of the CAR-T pi­o­neers’ play­book, Tes­sa is es­tab­lish­ing a joint ven­ture with Chi­na-Sin­ga­pore Guangzhou Knowl­edge City, which is ini­tial­ly putting down $40 mil­lion for a 13% stake with $40 mil­lion more to come in a sec­ond stage. The biotech, which now re­tains an 87% con­trol, is al­so rolling out its own con­tri­bu­tions in two phas­es, start­ing with $20 mil­lion and all its tech­nol­o­gy li­cense rights for Chi­na.

Bain’s biotech team has cre­at­ed a $1B-plus fund — with an eye to more Big Phar­ma spin­outs

One of the biggest investors to burst onto the biotech scene in recent years has re-upped with more than a billion dollars flowing into its second fund. And this next wave of bets will likely include more of the Big Pharma spinouts that highlighted their first 3 years in action.

Adam Koppel and Jeff Schwartz got the new life sciences fund at Bain Capital into gear in the spring of 2016, as they were putting together a $720 million fund with $600 million flowing in from external investors and the rest drawn from the Bain side of the equation. This time the external investors chipped in $900 million, with Bain coming in for roughly $180 million more.

They’re not done with Fund I, with plans to add a couple more deals to the 15 they’ve already posted. And once again, they’re estimating another 15 to 20 investments over a 3- to 5-year time horizon for Fund II.

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Search­ing for the next block­buster to fol­low Darza­lex, J&J finds a $150M an­ti-CD38 drug from part­ner Gen­mab

Now that J&J and Genmab have thrust Darzalex onto the regulatory orbit for first-line use in multiple myeloma, the partners are lining up a deal for a next-gen follow-on to the leading CD38 drug.


Janssen — J&J’s biotech unit — has its eyes on HexaBody-CD38, a preclinical compound generated on Genmab’s tech platform designed to make drugs more potent via hexamerization.


Genmab is footing the bill on studies in multiple myeloma and diffuse large B-cell lymphoma; once it completes clinical proof of concept, Janssen has the option to license the drug for a $150 million exercise fee. There’s also $125 million worth of milestones in play.

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Ab­b­Vie touts new da­ta for Hu­mi­ra suc­ces­sor; Gilead inks dis­cov­ery deal

→ Ab­b­Vie is tout­ing new pos­i­tive da­ta com­par­ing their ag­ing block­buster Hu­mi­ra with their hoped-for block­buster upadac­i­tinib. Over 48 weeks a larg­er pro­por­tion of pa­tients tak­ing the ex­per­i­men­tal drug ex­pe­ri­enced clin­i­cal re­mis­sion than in the con­trol arm with Hu­mi­ra. Their drug brought in $20 bil­lion last year, top­ping the scales in the num­ber 1 slot.

→ Gilead has turned to Van­cou­ver-based Ab­Cellera for its lat­est dis­cov­ery deal. Ab­Cellera will use its know-how in “sin­gle-cell screen­ing of nat­ur­al im­mune sources” to find an­ti­body can­di­dates for Gilead to pur­sue in the in­fec­tious dis­ease field. The deal in­cludes an up­front and mile­stones.